Af­ter tout­ing Gazy­va's po­ten­tial in lu­pus, Roche nabs 'break­through' ahead of PhI­II

Roche’s ef­fort to in­tro­duce its blood can­cer drug Gazy­va to the im­munol­o­gy space just got a boost from the FDA.

Reg­u­la­tors have grant­ed break­through des­ig­na­tion to the use of the CD20-tar­get­ing an­ti­body in lu­pus nephri­tis, a life-threat­en­ing man­i­fes­ta­tion of sys­temic lu­pus ery­the­mato­sus in the kid­ney. Up to 60% of all peo­ple with lu­pus — dis­pro­por­tion­ate­ly women — de­vel­op such in­flam­ma­tion in the kid­ney, the Lu­pus Foun­da­tion of Amer­i­ca es­ti­mates.

Im­muno­sup­pres­sants such as my­cophe­no­late mofetil or my­cophe­no­lic acid and cor­ti­cos­teroids are the cur­rent stan­dard of care, but Roche CMO San­dra Horn­ing not­ed that new treat­ment op­tions are need­ed. In a Phase II study, in­ves­ti­ga­tors showed that adding Gazy­va to the reg­i­men helped a larg­er pro­por­tion of pa­tients achieve com­plete re­nal re­sponse at one year.

A Phase III tri­al is slat­ed to be­gin next year.

While Gazy­va has pre­vi­ous­ly been po­si­tioned as a po­ten­tial suc­ces­sor to the ag­ing Rit­ux­an fran­chise, Roche re­cent­ly high­light­ed its po­ten­tial in lu­pus on its Phar­ma Day ear­li­er this week.

The ther­a­peu­tic piv­ot is part of a larg­er at­tempt to re­duce Roche’s de­pen­dence on the es­tab­lished — and high­ly prof­itable — on­col­o­gy port­fo­lio in fa­vor of nov­el drugs and tar­gets, Cowen an­a­lyst Steve Scala wrote in a note.

Roche made clear that mar­gins were not suf­fer­ing through the launch of new growth dri­vers Ocre­vus (mul­ti­ple scle­ro­sis) and Hem­li­bra (he­mo­phil­ia). Ro­bust up­take of both drugs has buoyed the topline while new pro­duc­tiv­i­ty ini­tia­tives saved costs, ex­em­pli­fied by suc­cess­ful launch­es de­spite adding no head count to sales forces. The com­pa­ny hopes to repli­cate this suc­cess when launch­ing in­to NMOSD with satral­izum­ab, IBD with etrolizum­ab, SMA with ris­diplam, Hunt­ing­ton’s with ASO-HTT, and lu­pus with Gazy­va.

The drug has been ap­proved to treat chron­ic lym­pho­cyt­ic leukemia and fol­lic­u­lar lym­phoma, with or fol­low­ing chemo. Its 2018 sales to­taled CHF 390 mil­lion (rough­ly $392 mil­lion).

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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Tri­fec­ta of sick­le cell dis­ease ther­a­pies ex­tend life ex­pectan­cy, but are not cost-ef­fec­tive — ICER

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.

UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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Ku­ra co-founder heads to Asian mul­ti-na­tion­al as biotech eyes the goal posts for lead drug

Six years after Kura Oncology snagged a farnesyl transferase inhibitor from J&J and leapt straight into clinical development, one of the biotech’s founders is leaving to start a new chapter in his career.

CMO and development chief Antonio Gualberto is exiting the company, and Kura — led by longtime biotech entrepreneur Troy Wilson — is on the hunt for a replacement. Wilson credited the CMO for some key biomarker work, including the discovery of the CXCL12 pathway as a target of their lead drug tipifarnib. Those biomarkers are being relied on to define the patient population most likely to benefit from the drug.

FDA waves Epizyme's $186K rare can­cer drug through to mar­ket — now get ready for the sec­ond act

After winning the hearts of the expert panel convened by the FDA despite a bleak in-house review and a checkered development history, Robert Bazemore has steered Epizyme to its first-ever OK for a rare cancer drug.

The approval in epithelioid sarcoma sets tazemetostat, now Tazverik, up nicely for a quick expansion to follicular lymphoma — a much bigger indication for which the biotech has just submitted an NDA.

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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